FDA Reverses Gene Therapy Rejection After Trump Official Ousted

A gene therapy for Huntington's disease has gained a new pathway toward FDA approval following the departure of Trump administration officials, particularly Vinay Prasad, who had rejected the treatment in a move that drew sharp criticism from within the regulatory community. A former FDA official described the rejection as "truly evil," highlighting the controversy surrounding the decision.

Huntington's disease is a progressive inherited neurological condition that typically emerges in middle age, causing gradual deterioration of brain nerve cells. The disease currently has no available treatments, and patients typically die in their 50s and 60s. UniQure's experimental gene therapy, AMT-130, represents a one-time treatment designed to reduce brain levels of huntingtin, the mutant protein responsible for the disease. Early trial data indicated the drug could slow disease progression by up to 75 percent, generating significant hope among patients and advocacy groups.

In 2024, the FDA initially signaled to UniQure that the company could pursue accelerated approval of AMT-130 without requiring a placebo control arm in its trial. This pathway reflected concern over the ethical implications of the proposed study design: delivering the gene therapy requires 10 to 12 hours of brain surgery, meaning a placebo control group would need to undergo sham surgery, potentially involving skull drilling. UniQure proceeded with an alternative approach, using external untreated patients as comparators instead of a control arm subjected to invasive procedures.

However, during Prasad's tenure as the FDA's head regulator for gene therapies, the agency reversed its position and demanded that UniQure conduct the sham surgeries as controls. This reversal prompted intense scrutiny and criticism from FDA insiders. With Prasad's departure and the removal of other Trump-appointed officials, the agency has now reconsidered its stance, opening the door for renewed regulatory discussions around the therapy's approval prospects.